Avidity Biosciences has released answers to frequently asked questions about their Managed Access Program (MAP) for individuals with Duchenne who are amenable to exon 44 skipping and meet clinical criteria. […]
A presentation with Dr. Doug Kerr, Chief Medical Officer at Dyne Therapeutics. Dr. Kerr shares topline results from the DELIVER clinical trial, along with new long-term data showing sustained benefits […]
In 2018, CureDuchenne was the only Duchenne-focused organization to invest in Avidity Biosciences, recognizing the potential of their novel RNA-targeting approach for individuals with Duchenne muscular dystrophy long before it reached clinical trials. […]
PepGen has ended clinical trials of PGN-EDO51, their investigational therapy for Duchenne patients amenable to skipping exon 51, as PGN-EDO51 failed to achieve target dystrophin levels in their CONNECT1 trial. The company intends […]
CureDuchenne provided early funding to Entrada Therapeutics. Entrada has received regulatory authorization in Europe to initiate a Phase 1/2 clinical trial of ENTR-601-44 in ambulatory individuals with Duchenne amenable to […]
Wave Life Science Exon Skipping in Duchenne Wave Life Sciences: Update on 48-week Data from FORWARD-53 Exon Skipping Study: An update on Wave’s Phase 2 FORWARD-53 trial of WE-N531, Wave’s […]
Duchenne muscular dystrophy (DMD) – An update on the DELIVER trial of DYNE-251 in males with DMD mutations amenable to exon 51 skipping. WATCH HERE
Wave Life Sciences Exon-skipping Trial Wave Life Sciences has shared new data from its ongoing FORWARD-53 clinical trial, evaluating the investigational exon-skipping therapy WVE-N531 for individuals with Duchenne muscular dystrophy […]
Entrada trial skipping exon 45 CureDuchenne provided early funding to Entrada Therapeutics, who has announced that they received authorization in the United Kingdom (UK) to initiate a Phase 1/2 multiple […]
